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A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

NCT05791864 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2025-12-18

No results posted yet for this study

Summary

This is a first-in-human, open-label, single ascending dose study of TTX-381 for the treatment of ocular manifestations of CLN2 (Batten disease).

Conditions

  • Neuronal Ceroid Lipofuscinosis Type 2

Interventions

GENETIC

TTX-381

One time subretinal dose in study eye

Sponsors & Collaborators

  • Tern Therapeutics, LLC

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SEQUENTIAL

Eligibility

Min Age
12 Months
Max Age
84 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-05-17
Primary Completion
2026-07-30
Completion
2031-07-30
FDA Drug
Yes

Countries

  • Germany
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05791864 on ClinicalTrials.gov